A Post-Authorisation Safety Study Patient Registry of Patients With Neuroblastoma Being Treated With Dinutuximab Beta
Rekrutierend
NCT-Nummer:
NCT04253015
Studienbeginn:
September 2019
Letztes Update:
07.02.2024
Wirkstoff:
-
Indikation (Clinical Trials):
Neuroblastoma
Geschlecht:
Alle
Altersgruppe:
Alle
Phase:
-
Sponsor:
EusaPharma (UK) Limited
Collaborator:
United BioSource, LLC
Studienleiter
Jose-Luis Garcia Study DirectorEUSA Pharma (UK) Limited
Kontakt
Jose-Luis Garcia Kontakt: Phone: +34 663 36 34 24 E-Mail: PASS@eusapharma.com» Kontaktdaten anzeigen
Studienlocations (3 von 13)
Wien, Austria Lille France Marseille Paris Villejuif Berlin Germany Greifswald Mecklenburg-Vorpommern Deutschland Genova Italy Kraków Poland Valencia Spain Newcastle Upon Tyne United Kingdom Birmingham Southampton
Charité Berlin 13353 Berlin (Berlin) GermanyRekrutierend » Google-Maps Ansprechpartner: Angelika Eggert, Dr Phone: + 49 30 450 566 808 E-Mail: angelika.eggert@charite.deHeidi Deubzer, Dr Phone: + 49 30 450 566 808 E-Mail: heidi.deubzer@charite.de» Ansprechpartner anzeigen Interdisziplinäres Brustzentrum der Universitätsmedizin Greifswald Ferdinand-Sauerbruch-Straße 17475 Greifswald Greifswald (Mecklenburg-Vorpommern) DeutschlandRekrutierend » Google-Maps Ansprechpartner: Holger Lode, Dr Phone: +49 3834 86-6301 E-Mail: holger.lode@uni-greifswald.deKaroline Ehlert, Dr Phone: +49 3834 86-6301 E-Mail: ehlertk68@uni-greifswald.de» Ansprechpartner anzeigen St. Anna Kinderkrebsforschung 1090 Wien, AustriaAktiv, nicht rekrutierend » Google-Maps Centre Oscar Lambret 59000 Lille FranceRekrutierend » Google-Maps Ansprechpartner: Fabienne Dumont, Mr Phone: +33 3.20.29.59.35 E-Mail: f-dumont@o-lambret.fr» Ansprechpartner anzeigen Hôpital de la Timone, Hôpital des Enfants 13385 Marseille FranceRekrutierend » Google-Maps Ansprechpartner: Sylvie Abed Phone: 00 33 4 91 38 68 21» Ansprechpartner anzeigen Institut Curie 75005 Paris FranceAktiv, nicht rekrutierend » Google-Maps Institut Gustave Roussy 94805 Villejuif FranceRekrutierend » Google-Maps Ansprechpartner: Imene Hazem, Mr Phone: 0033 1 42 11 50 20 E-Mail: Imene.HEZAM@gustaveroussy.frFanny Prenois, Mrs Phone: 0033 1 42 11 50 20 E-Mail: Fanny.PRENOIS@gustaveroussy.fr» Ansprechpartner anzeigen IRCCS Istituto Giannina Gaslini 16147 Genova ItalyRekrutierend » Google-Maps Ansprechpartner: Sabrina Zanardi Phone: +39.010.5636.3461 E-Mail: SabrinaZanardi@gaslini.org» Ansprechpartner anzeigen Uniwersytecki Szpital Dziecięcy 30-663 Kraków PolandRekrutierend » Google-Maps Ansprechpartner: Aleksandra Wieczore, Dr Phone: (+48) 12 3339392 E-Mail: a.wieczorek@uj.edu.plWalentyna Balwierz, Dr Phone: (+48) 12 658 20 11 Phone (ext.): 4321 E-Mail: walentyna.balwierz@uj.edu.pl» Ansprechpartner anzeigen Hospital Universitario y Politecnico La Fe Avenida Fernando Abril Martorell 46026 Valencia SpainRekrutierend » Google-Maps Ansprechpartner: Desiree Ramal Phone: +34-638902615» Ansprechpartner anzeigen The Newcastle upon Tyne Hospitals NHS Foundation Trust NE1 4LP Newcastle Upon Tyne United KingdomRekrutierend » Google-Maps Ansprechpartner: Geoff Bell Phone: 0191 282 1337 E-Mail: Geoff.Bell@nuth.nhs.ukLinda Coates Phone: 0191 2829964 E-Mail: Linda.Coates7@nhs.net» Ansprechpartner anzeigen Birmingham Children's Hospital B4 6NH Birmingham United KingdomAktiv, nicht rekrutierend » Google-Maps University Hospital Southampton SO16 6YD Southampton United KingdomRekrutierend » Google-Maps Ansprechpartner: Ruth Lawrence Phone: 02381206334 E-Mail: Ruth.Lawrence@uhs.nhs.ukAlice Johnson Phone: 02381206334 E-Mail: alice.johnson@uhs.nhs.uk» Ansprechpartner anzeigen Alle anzeigen
Detailed Description: Rationale and Background: Neuroblastoma, is the most common extra-cranial solid tumour in children. Most patients with neuroblastoma are diagnosed under the age of 5 years and most present with metastatic disease and/or high-risk features. Despite the introduction of novel treatment strategies, including high-dose chemotherapy followed by autologous stem cell transplantation (ASCT), the outcome of these patients remains poor. Dinutuximab beta is a chimeric monoclonal immunoglobulin G 1 (IgG1) antibody that is specifically directed against the carbohydrate moiety of disialoganglioside antigen (GD2), which is overexpressed on neuroblastoma cells. By binding to neuroblastoma cells, dinutuximab beta can induce both complement dependent cytotoxicity (CDC) and antibody dependent cell-mediated cytotoxicity (ADCC). The efficacy of dinutuximab beta has been evaluated in a randomised controlled trial comparing the administration of dinutuximab beta with or without interleukin 2 (IL-2) in the first-line treatment of patients with high-risk neuroblastoma and in two singlearm studies in the relapsed/refractory setting. The efficacy and safety of dinutuximab beta will further be evaluated in this registry that will provide information on survival, pain severity and incidence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events, hypersensitivity reactions and long-term safety. Study Design: This is a non-interventional, multi-national, observational, prospective patient registry of patients with high-risk neuroblastoma being treated with the monoclonal antibody dinutuximab beta. The efficacy and safety of dinutuximab beta will further be evaluated in this registry that will provide information on survival, pain severity and incidence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events, hypersensitivity reactions and long-term safety. Research Questions and Objectives: Primary objectives: - To assess pain severity and use of analgesics during the period of first dose of dinutuximab beta to end of last 35 day course of the 5th cycle of treatment - To assess the incidence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events and hypersensitivity reactions - To assess the long term safety profile Secondary objectives: - Progression free survival (PFS) in patients treated with dinutuximab beta. - Event Free Survival (EFS) in patients treated with dinutuximab beta - Overall survival (OS) in patients treated with dinutuximab beta Population: Patients diagnosed with high-risk neuroblastoma who are starting treatment with dinutuximab beta in the standard clinical practice setting or participating in a clinical trial where dinutuximab beta is provided according to the indication as per the country/regional marketing authorisation, provide consent/assent and are willing to be followed up for up to 10 years. Study Size: It is planned to enroll a sufficient number of patients (estimated at 125) such that 100 patients will have completed all five treatment courses of dinutuximab beta. It is anticipated that this will result in 40-50 patients who are progression free at 10 years. Data Sources: Data will be collected from physicians using an electronic data capture (EDC) system. The electronic case report forms (eCRFs) will be designed to gather data from the medical records at baseline, during treatment and at normal clinical practice follow up visits. Data Analysis: The safety analysis set, containing all patients treated with at least one dose of dinutuximab beta will be considered for safety and efficacy analyses. All baseline, treatment period and follow up characteristics will be summarized using descriptive statistics. Endpoints addressing primary and secondary analysis will include 95% confidence intervals (CIs) including the Clopper Pearson method for binomial, log-log transform for survival. OS, PFS and EFS will be analysed using Kaplan-Meier methods. Variables: Baseline (prior to start of treatment): Demographics, clinical trial participation, neuroblastoma disease history, and presence or absence of neurotoxicity, visual impairment, and cardiovascular abnormality. Treatment period (up to end of last 35 day course of 5th cycle of treatment): Dosing regimen, total cumulative amount of dinutuximab beta per course, concomitant medications during course (IL-2, retinoic acid and/or antihistamines), daily analgesics (opioids, gabapentin/ pregabalin and/or non-opioid analgesics and other neuropathic pain treatments), daily pain assessment during infusion of dinutuximab beta, occurrence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events, and hypersensitivity reactions, adverse events (AEs)/serious adverse events (SAEs) treatment interruptions and discontinuations, progression of disease, date and cause of death, reason for study withdrawal (if applicable) Follow up (from end of last 35 day course of 5th cycle of treatment): Status of neurotoxicity, visual impairment, cardiovascular events, (resolved, not resolved), SAEs and adverse drug reaction (ADRs), progression of disease, date and cause of death, reason for study withdrawal (if applicable).
Inclusion Criteria: Patients meeting the following criteria will be considered for inclusion into the registry: - Patients diagnosed with high-risk neuroblastoma and starting treatment with commercially available dinutuximab beta OR - Patients diagnosed with high-risk neuroblastoma and starting treatment with dinutuximab beta in a clinical trial where dinutuximab beta is provided according to the country/regional marketing authorisation AND - Appropriate consent/assent has been obtained for participation in the registry with a willingness to be followed up for up to 10 years.Exclusion Criteria: Patient will not be eligible for inclusion if the following criterion applies: - Patients commencing dinutuximab beta within a clinical trial where the product is being provided outside of the country/regional marketing authorisation OR - Appropriate consent/assent has not been obtained for participation in the registry or patient/legal representative is not willing for the patient be followed up for up to 10 years.
Primary outcome: 1. Assessment of the severity of pain experienced by participants during treatment with dinutuximab beta (Time Frame - First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)):Assessment of pain severity experienced by participants during the period of first dose of dinutuximab beta to the end of last 35 day course of 5th cycle of treatment 2. Number of participants using analgesics during treatment with dinutuximab beta (Time Frame - First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)):Use of analgesics during the period of first dose of dinutuximab beta to end of last 35 day course of 5th cycle of treatment 3. Incidence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events and hypersensitivity reactions (Time Frame - First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)):Incidence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events and hypersensitivity reactions up to the end of the last 35 day course of 5th cycle of treatment 4. Number of participants experiencing serious adverse events (SAEs) and adverse drug reactions (ADRs) during treatment with dinutuximab beta (Time Frame - First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)):Number of participants experiencing serious adverse events (SAEs) and adverse drug reactions (ADRs) following the end of the last 35 day course of 5th cycle of treatment Secondary outcome: 1. Overall Survival (OS) (Time Frame - First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)):Overall Survival (OS) following the end of the last 35 day course of 5th cycle of treatment 2. Progression free survival (PFS) (Time Frame - First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)):Progression free survival (PFS) following the end of the last 35 day course of 5th cycle of treatment 3. Event Free Survival (EFS) (Time Frame - First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)):Event Free Survival (EFS) following the end of the last 35 day course of 5th cycle of treatment
Data-collection:Data will be collected on dose, total cumulative amount of dinutuximab beta per course, dose interruptions, dose discontinuations, prophylactic treatment, use of all concomitant analgesia, assessments of pain, and occurrence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events and hypersensitivity reactions and other AEs.
Quelle: ClinicalTrials.gov
Sie können folgenden Inhalt einem Kollegen empfehlen:
"A Post-Authorisation Safety Study Patient Registry of Patients With Neuroblastoma Being Treated With Dinutuximab Beta"
Bitte tragen Sie auch die Absenderdaten vollständig ein, damit Sie der Empfänger erkennen kann.
Die mit (* ) gekennzeichneten Angaben müssen eingetragen werden!